Open-label trial of ranolazine for the treatment of paramyotonia congenita.

ABSTRACT:

Introduction

Paramyotonia congenita (PMC) is a nondystrophic myotonic disorder that is believed to be caused by a defect in Na_v 1.4 sodium channel inactivation. Ranolazine, which acts by enhancing slow inactivation of sodium channels, has been proposed as a therapeutic option, but in vivo studies are lacking.

Methods

We conducted an open-label, single-center trial of ranolazine to evaluate efficacy and tolerability in patients with PMC. Subjective symptoms of stiffness, weakness, and pain as well as clinical and electrical myotonia were evaluated. Baseline measures were compared with those after 4 weeks of treatment with ranolazine.

Results

Ranolazine was tolerated well without any serious adverse events. Both subjective symptoms and clinical myotonia were

SUBMITTER: Lorusso S 

PROVIDER: S-EPMC6340713 | biostudies-literature | 2019 Feb

REPOSITORIES: biostudies-literature